用皮改性释放硬囊治疗先天性上腺增生症患者的长期结局
Wiebke Arlt1,2, Aude Brac de la Perrière3, Angelica L Hirschberg4,5
1MRC Laboratory of Medical Sciences (MRC LMS), London W12 0HS, United Kingdom.
European journal of endocrinology
|June 27, 2025
概括
修正释放皮松囊 (MRHC) 改善了先天性上腺增生 (CAH) 的生化控制,并减少了长期的皮松剂量. 这种治疗也显示出对生育能力和上腺危机发生率较低的有希望的结果.
科学领域:
- 内分泌学 在内分泌学.
- 药理学 药理学是指药理学的学科.
- 遗传学 是一个遗传学.
背景情况:
- 先天性上腺增生 (CAH) 是一组影响上腺的遗传性疾病.
- 皮改性释放硬囊 (MRHC) 旨在模仿身体的自然皮质醇节奏.
- MRHC的目标是通过提供更生理性的皮质醇替代物来改善CAH的管理.
研究的目的:
- 评估MRHC在经典CAH患者的长期安全性和耐受性.
- 评估MRHC在长期改善CAH的生物化学控制中的有效性.
- 调查MRHC对生育能力的影响和上腺危机的发生率.
主要方法:
- 采用了一种开放式的后续研究设计.
- 91名患有经典CAH的患者参与了研究,治疗时间中位数为4年.
- 监测了皮松剂量,类固醇疾病标志物 (17OHP,A4),怀孕率和上腺危机发生率.
主要成果:
- 降低了皮的剂量并长期稳定,改善了CAH的生物化学控制 (17OHP和androstenedione水平).
- 很大一部分患者的疾病标志物水平在正常范围内或以下.
- 在女性和男性中观察到生育率,上腺危机的发生率低于以前报告的.
结论:
- 治疗MRHC导致持续降低皮的剂量,并改善了CAH患者的生化控制.
- 生物化学监测是可靠的,单个血液样本在09:00和13:00之间取.
- MRHC显示出有利的安全性,减少了上腺危机发生率,对生育能力产生了积极影响.
相关概念视频
Adrenal Gland Disorders
1.9K
Adrenal gland disorders manifest when the production of adrenal hormones deviates from the norm, resulting in either excessive or insufficient concentrations.
Adrenal insufficiency, characterized by insufficient cortisol and aldosterone production, leads to conditions like Addison's disease. This disorder, affecting the adrenal cortex, exhibits symptoms such as skin bronzing, dehydration, low blood pressure, fatigue, and weight loss. Congenital adrenal hyperplasia, a genetic ailment causing...
Adrenal insufficiency, characterized by insufficient cortisol and aldosterone production, leads to conditions like Addison's disease. This disorder, affecting the adrenal cortex, exhibits symptoms such as skin bronzing, dehydration, low blood pressure, fatigue, and weight loss. Congenital adrenal hyperplasia, a genetic ailment causing...
1.9K
Hormones of the Adrenal Glands
2.9K
Adrenal hormones play a pivotal role in maintaining the body's electrolyte balance and orchestrating responses to stress, showcasing the intricate functions of the adrenal cortex and medulla.
The adrenal cortex, a powerhouse of hormone synthesis, generates over two dozen corticosteroid hormones. The zona glomerulosa produces mineralocorticoids, exemplified by aldosterone, influencing the electrolyte composition of body fluids. The synthesis of glucocorticoids such as cortisol and...
The adrenal cortex, a powerhouse of hormone synthesis, generates over two dozen corticosteroid hormones. The zona glomerulosa produces mineralocorticoids, exemplified by aldosterone, influencing the electrolyte composition of body fluids. The synthesis of glucocorticoids such as cortisol and...
2.9K
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
208
Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
208
Antiasthma Drugs: Inhaled Corticosteroids and Glucocorticoids
399
Inhaled corticosteroids (ICS) are anti-inflammatory drugs used primarily in treating persistent asthma and providing long-term maintenance. They target the bronchial mucosa, the lining of the airways, to control inflammation, a critical factor in asthma progression and exacerbation.
ICS work through a multifaceted mechanism of action. They suppress the inflammatory response caused by the proliferation of TH cells. They also reduce the transcription of the IL-2 gene, which is involved in the...
ICS work through a multifaceted mechanism of action. They suppress the inflammatory response caused by the proliferation of TH cells. They also reduce the transcription of the IL-2 gene, which is involved in the...
399
COPD: Management Using Bronchodilators and Corticosteroids
324
Chronic obstructive pulmonary isease (COPD) involves a group of progressive lung disorders characterized by persistent airflow limitation and chronic respiratory symptoms. Asthma-COPD Overlap Syndrome (ACOS), encompassing features of both asthma and Chronic obstructive pulmonary disease (COPD), is a group of progressive lung disorders that includes chronic bronchitis, emphysema, and refractory (non-reversible) asthma. ACOS leads to complex clinical presentations that combine the inflammatory...
324
Hypothalamic-Pituitary Axis
61.9K
The response to stress—be it physical or psychological, acute or chronic—involves activation of the Hypothalamic-Pituitary-Adrenal (HPA) axis. The HPA axis is part of the neuroendocrine system because it involves both neuronal and hormonal communication. Its function is to regulate homeostatic systems—metabolic, cardiovascular, and immune—providing the necessary means to respond to a stressor.
61.9K


